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Phase 2 N=59 Randomized Double-blind Treatment

Investigating Efficacy and Safety of Once-weekly NNC0195-0092 Treatment Compared to Daily Growth Hormone Treatment (Norditropin® FlexPro®) in Growth Hormone Treatment naïve Pre-pubertal Children With Growth Hormone Deficiency

Growth Hormone Disorder · Growth Hormone Deficiency in Children

Enrolled (actual)
59
Serious AEs
8.1%
Results posted
Jan 2026
Primary outcome: Primary: Height Velocity (HV) (cm/Year) During the First 26 Weeks of Treatment, Measured as Standing Height With Stadiometer — 11.35; 7.96; 10.92; 12.88 Centimetre per year (Cm/year)

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
somapacitan (Drug); Norditropin® FlexPro® pen (Drug)
Age
Pediatric · 2+ yrs
Sex
All
Sponsor
Novo Nordisk A/S
Primary completion
Sep 2024

Outcome Measures

OutcomeResultp-value
PRIMARY
Height Velocity (HV) (cm/Year) During the First 26 Weeks of Treatment, Measured as Standing Height With Stadiometer
11.35; 7.96; 10.92; 12.88
PRIMARY
Cohort II and Cohort III - Adverse Events Rate, Including Injection Site Reactions in Children With GHD.
1047; 115.4; 196.9
SECONDARY
Cohort I: Change in Height Standard Deviation Score (HSDS)
0.66; 0.31; 0.63; 0.89; 0.98; 0.59
SECONDARY
Cohort I: Change in Height Velocity Standard Deviation Score (HVSDS)
9.02; 4.93; 7.27; 10.01; 7.41; 4.72
SECONDARY
Cohort I: Adverse Events Rate, Including Injection Site Reactions
247.7; 116.3; 210.4; 311.5; 364.8; 163.1
SECONDARY
Cohort I: Occurrence of Anti-NNC0195-0092 and Anti-hGH Antibodies
0; 2; 2; 2; 6; 1
SECONDARY
Change in Insulin-like Growth Factor I (IGF-I) Standard Deviation Score (SDS)
1.86; 0.95; 1.99; 3.00; 1.67; 0.98
SECONDARY
Change in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) Standard Deviation Score (SDS)
1.58; 0.86; 1.59; 1.45; 0.95; 1.05
SECONDARY
Height Velocity (HV) (cm/Year) at Weeks 52 (Derived From Standing Height)
9.8; 7.8; 9.7; 11.5
SECONDARY
Bone Age Progression vs. Chronological Age Ratio
0.577; 0.570; 0.651; 0.705
SECONDARY
Serum Somapacitan Concentrations
18.381; 44.563; 169.320
SECONDARY
Changes in Emotional Well-being Score, Physical Health Score, Social Well-being Score and Total Score in TRIM-CGHD-O (Treatment Related Impact Measure - Child Growth Hormone Deficiency- Observer)
4.3; -4.0; -2.8; -11.6; -1.9; 0.9
SECONDARY
Total Score of TB-CGHD-O (The Treatment Burden Measure - Child Growth Hormone Deficiency - Observer)
14.8; 14.0; 10.2; 6.8; 12.3; 13.7
SECONDARY
Total Score of TB-CGHD-P (The Treatment Burden Measure - Child Growth Hormone Deficiency - Parent/Guardian)
26.9; 24.6; 17.5; 18.8; 23.6; 19.6

Summary

This trial is conducted globally. The aim of the trial is to investigate efficacy and safety of once-weekly NNC0195-0092 treatment compared to daily growth hormone treatment (Norditropin® FlexPro®) in growth hormone treatment naïve pre-pubertal children with growth hormone deficiency. The main trial period will consist of 26 weeks of treatment, followed by a 26 week extension period.

Eligibility Criteria

Inclusion Criteria

  • Boys: Tanner stage 1 for pubic hair and testis volume below 4 ml , age at least 2 years and 26 weeks and below or equal to 10.0 years at screening
  • Girls: Tanner stage 1 for breast development (no palpable glandular breast tissue) and pubic hair, age at least 2 years and 26 weeks and below or equal to 9.0 years at screening
  • Confirmed diagnosis of GHD (growth hormone deficiency) within 12 months prior to screening as determined by two different GH (growth hormone) stimulation tests, defined as a peak GH level of below or equal to 7.0 ng/ml. For children with three or more pituitary hormone deficiencies only one GH stimulation test is needed
  • No prior exposure to GH therapy and/or IGF-I (insulin-like growth factor I) treatment
  • Height of at least 2.0 standard deviations below the mean height for chronological age (CA) and gender according to the standards of Centers for Disease Control and Prevention 2-20 years: Girls/Boys stature-for-age and weight-for-age percentiles CDC at screening
  • Annualized height velocity (HV) below the 25th percentile for CA (chronological age) and gender or below -0.7 SD (standard deviation) score for CA and sex, according to the standards of Prader calculated over a time span of minimum 6 months and maximum 18 months

Exclusion Criteria

  • Any clinically significant abnormality likely to affect growth or the ability to evaluate
  • growth with standing measurements: Chromosomal aneuploidy and significant gene mutations causing medical "syndromes" with short stature, including but not limited to Turner syndrome, Laron syndrome, Noonan syndrome, or absence of GH receptors. Congenital abnormalities (causing skeletal abnormalities), including but not limited to Russell-Silver Syndrome, skeletal dysplasias. Significant spinal abnormalities including but not limited to scoliosis, kyphosis and spina bifida variants
  • Children born small for gestational age (SGA - birth weight and/or birth length below-2 SD for gestational age)
  • Concomitant administration of other treatments that may have an effect on growth, including but not limited to methylphenidate for treatment of attention deficit hyperactivity disorder (ADHD)
  • Prior history or presence of malignancy and/or intracranial tumour
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02616562). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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